Greater Philadelphia Q4 2022 News Recap – December was a Pivotal Month for Cellicon Valley Companies

Cellicon Valley companies closed out 2022 with a roar. Madrigal Pharmaceuticals announced a breakthrough in NASH that could result in the first-approved drug for that disease. Galera Therapeutics closed out December with plans to seek approval for its severe oral mucositis therapy.

BioBuzz takes a look back at some of the last minute pivotal events for Philadelphia companies. 

Breakthrough in NASH

Madrigal Pharmaceuticals received an early present from Saint Nicholas in the form of a major breakthrough in nonalcoholic steatohepatitis (NASH), one of the hottest areas of research across the biopharmaceutical industry.

In December, Madrigal announced positive topline data from its Phase III MAESTRO-NASH trial evaluating resmetirom, a liver-directed selective thyroid hormone receptor agonist. The study met its primary endpoint as well as key secondary endpoints.

“These pivotal Phase III results demonstrate the potential for resmetirom to help patients achieve improvement in both the underlying steatohepatitis that drives this disease and the resulting fibrosis that is associated with progression to cirrhosis and its complications. The topline data also reinforce our confidence in the safety and tolerability profile of resmetirom, Madrigal Chief Medical Officer Becky Taub said in a statement.

The data from the MAESTRO trial, as well as data from other studies will provide a strong foundation for the NDA.

With the positive data in hand, Madrigal could be the first company to the finish line with a treatment specifically for NASH, an advanced form of fatty liver disease. The company said it will file a New Drug Application for resmetirom in the first half of 2023. Madrigal anticipates the filing will be under the FDA’s accelerated approval program.

Headway on Universal Flu Vaccine

Researchers at the University of Pennsylvania are making headway on an mRNA-based universal flu vaccine.

The experimental vaccine is designed to protect the immune system against all 20 subtypes of both influenza A and B. The wide swath of protection could prevent infection from most known flu viruses. 

In November, reports showed the vaccine was found to protect mice and ferrets against severe influenza. The next step is to study the vaccine in humans. If all goes well in the trial, the UPenn-developed vaccine could be available for use within the next few years.

Galera Heads to FDA with Severe Oral Mucositis Treatment

With positive data in hand, Galera Therapeutics will seek approval for avasopasem, a potential therapeutic for radiotherapy-induced severe oral mucositis (SOM) in patients with head and neck cancer.

Data showed avasopasem more than halved the median number of days a patient suffered from SOM. Additionally, clinical data showed avasopasem generated clinically meaningful reductions in severity of SOM compared to placebo. 

SOM is characterized by the inability to eat solid food or drink liquids and may require the surgical placement of feeding tubes to maintain nutrition and hydration. There are no FDA-approved drugs to reduce SOM for these patients.

The FDA granted avasopasem both Fast Track and Breakthrough Therapy designations for the reduction of SOM induced by radiotherapy.

Heading into the Clinic

Startup iECURE secured $65 million in financing and will use the funds to take its lead asset for Ornithine Transcarbamylase (OTC) deficiency into the clinic.

iECURE’s lead program is aimed at OTC deficiency, the most common urea cycle disorder. OTC is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. 

The gene editing company is using in vivo gene insertion to insert full, healthy donor genes into the liver of a juvenile patient. GTP-506 is designed to insert a donor OTC gene into the liver of an infant in order to deliver the necessary genetic correction so the child can live a full and healthy life,

Key M&A Activity

In a last minute deal that links the BioHealth Capital Region with Philadelphia, Kite, a Gilead Sciences company, bolstered its cell therapy capabilities with the acquisition of Carl June-founded Tmunity Therapeutics.

Kite announced in December that Tmunity’s next-generation CAR-T therapies and technology will complement its own cell therapy research capabilities. Kite gains multiple clinical and preclinical programs, as well as Tmunity’s “armored” CAR-T technology platform. Kite believes this technology can provide CAR-Ts with the capability to enhance anti-tumor activity. The company announced the platform can also improve manufacturing.

Also this year, Pennsylvania-based Idera snapped up N.C.-based Aceragen to support its rare disease portfolio. The acquisition brings Aceragen’s pipeline that addresses rare, orphan pulmonary and rheumatic diseases for which there are limited or no available treatments into Idera’s portfolio.

Key assets included in the deal are ACG-701 and ACG-801, experimental treatments for cystic fibrosis and melioidosis patients, and Farber disease respectively.

Philadelphia Real Estate News

JLL finished out the year with a $290 million construction loan for a state-of-the-art research facility in downtown Philadelphia on the Drexel University campus. The 11-story building will provide a mixed use for multiple life sciences companies, as well as retail space.

The site in Philadelphia’s University City submarket will provide critical lab infrastructure, chemical storage space and ph neutralization capabilities. Nearly half of the space in the new facility is already spoken for. SmartLabs and Drexel University have pre-leased 45% of the lab space. 

Also this year, Verismo Therapeutics opened a new Research and Development Center at CIC Philadelphia to advance the development to boost the R&D and process development capabilities of the Philadelphia-based company and advance its novel KIR-CAR platform.

The CIC Philadelphia-based lab space will serve as one of Verismo’s in-house translational research and process development work. Work conducted at the site will support the ongoing activities for the SynKIR-110 clinical trial and future pipeline products including SynKIR-210 and SynKIR-310.